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Abiraterone Acetate in Children with Classic 21-Hydroxylase Deficiency

The purpose of this study is to (1) determine the minimum effective dose of abiraterone acetate that normalizes androstenedione levels in prepubertal children with CAH secondary to 21-hydroxylase deficiency (Phase 1 trial), and (2) assess the utility of abiraterone acetate in prepubertal children with CAH as adjunctive therapy to minimize excessive androgen secretion and allow more physiological glucocorticoid replacement (Phase 2 trial).

Adrenas CAH (Congenital Adrenal Hyperplasia) Study

This is a Phase 1/2, first-in-human, open-label, dose-escalation study designed to evaluate the safety, tolerability, and efficacy of BBP-631 administered to up to 25 adult participants diagnosed with classic congenital adrenal hyperplasia (CAH) (simple virilizing or salt-wasting, Group 1) or with classic salt-wasting CAH (Group 2) due to 21-hydroxylase deficiency (21-OHD) and who are monitored for 24 weeks post-treatment. All participants who receive BBP-631 will be followed for an additional 4.5 years for safety and efficacy in a separate long term follow up protocol (Study CAH-399). In total, all participants will be followed for at least 5 years after the date of treatment with BBP-631.

CTLA (Abatacept) Prevention (TN18)

Abatacept is given as an investigational study drug to relatives of someone with type 1 diabetes who have positive diabetes antibodies in an attempt to prevent or delay the onset of type 1 diabetes. See TrailNet.org website for more details.

Diurnal Study

This study is a randomized, double-blind, active-controlled, titrated, parallel arm, multicenter study. It will compare the efficacy, safety and tolerability of twice daily Chronocort with twice daily IRHC (Cortef®) over a randomized treatment period of up to 52 weeks in participants aged 16 years and over with known classic CAH due to 21-hydroxylase deficiency. The primary efficacy assessment of biochemical responder rate and the key secondary assessments of dose responder rate and mean total daily dose will be assessed after 52 weeks of randomized treatment.

DSRD Clinical Trial

The first Phase IIb clinical trial in Down syndrome regression disorder (DSRD). An open-label, randomized study comparing the safety and efficacy of lorazepam, intravenous immunoglobulin (IVIg) and the Janus Kinase inhibitor (JAK inhibitor), tofacitinib.

HIGH DOSE ERYTHROPOIETIN FOR ASPHYXIA AND ENCEPHALOPATHY (HEAL)

1) To determine if 5 doses of Epo (Erythropoietin) 1000 U/kg (birth weight) intravenous (IV) reduces the rate of death or neurodevelopmental impairment (mild, moderate, or severe) at 24 months of age. 

2) To assess safety of Epo. 

3) To determine whether Epo decreases the severity of HIE-induced brain injury as evidenced by early MRI and plasma biomarkers of brain injury.